Condition (MeSH)
Genetic Diseases, X-Linked
ClinicalTrials.gov lists 1,704 trials for Genetic Diseases, X-Linked or a narrower condition beneath it. National Institute of Allergy and Infectious Diseases (NIAID) has the most among industry and government lead sponsors, with 39.
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Trials per phase
1 more section with rows
40 of the 1,704 trials are tagged with Genetic Diseases, X-Linked itself; the rest are tagged with a narrower condition beneath it.
When did Genetic Diseases, X-Linked trials start? (37)
Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year. 8 trials are registered to start in 2027 or later, and not listed here.
Trials over time, by phase
- Phase 1
- Phase 1/2
- Phase 2
- Phase 2/3
- Phase 3
- Phase 4
- Not applicable
- Observational
Top of scale: 104
Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.
| Year | Trials started |
|---|---|
| 2026 (partial year) | 75 |
| 2025 | 65 |
| 2024 | 103 |
| 2023 | 104 |
| 2022 | 101 |
| 2021 | 94 |
| 2020 | 80 |
| 2019 | 82 |
| 2018 | 100 |
| 2017 | 76 |
Showing 10 of 37 earlier years.
Forecast of the full-year count, planned starts included, not rows of this table: about 94 trials starting in 2026 and about 85 in 2027, the average of the 24 months through September, counted as registered and carried forward, with about 0.7 times the starts already registered for the rest of 2026 added. The rows of this table count trials registered so far with a start date in the year, planned starts included; the forecast looks ahead to the count for the whole year, so it is not the same measure. In backtests on conditions of this size, forecasts made part-way through the year were typically off by about 15 percent and ran about 1 percent high against the count later registered; forecasts made before the year began were off by about 26 percent and ran about 20 percent low against the count later registered. The part-way figures are from forecasts that used a multiple fitted only on earlier years. These figures rest on a few years of history (target years from 2018 to 2025, varying by figure), place each condition as it is mapped today rather than as it was then, and score against a count for the newest year that was still being revised. Only condition series were scored.
Prediction, not a registry value: median of 36 months from start to primary completion across 156 trials still running at the 2026-10-09 snapshot; registered median of 29 months across 1,011 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Genetic Diseases, X-Linked trials in? (9)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 186 |
| Phase 1/2 | 159 |
| Phase 2 | 248 |
| Phase 2/3 | 51 |
| Phase 3 | 180 |
| Phase 4 | 55 |
| Not applicable | 213 |
| Observational | 593 |
| Expanded access | 19 |
How many Genetic Diseases, X-Linked trials are recruiting? (12)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 49 |
| Recruiting | 232 |
| Enrolling by invitation | 38 |
| Active, not recruiting | 103 |
| Available | 9 |
Showing 5 of 12 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Genetic Diseases, X-Linked trials? (13)
Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.
| Sponsor | Trials |
|---|---|
| National Institute of Allergy and Infectious Diseases (NIAID) | 39 |
| Novo Nordisk A/S | 36 |
| Pfizer | 29 |
| Sarepta Therapeutics, Inc. | 27 |
| Shire | 27 |
Showing 5 of 13 sponsors.
Which drugs are being tested for Genetic Diseases, X-Linked? (11)
Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.
| Drug | Trials |
|---|---|
| REPLAGAL | 20 |
| Migalastat | 20 |
| AGALSIDASE BETA (GZ419828) | 20 |
| BENEFIX | 18 |
| Busulfan | 16 |
Showing 5 of 11 drugs.
Interventions as named in the registry (4)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Alemtuzumab s.c. | 15 |
| SQ Fludarabine | 14 |
| Total Body Irradiation | 10 |
| No treatment given | 9 |
These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.
Which serious adverse events do Genetic Diseases, X-Linked trials report? (10)
Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.
| Adverse event | Subjects affected | Trials |
|---|---|---|
| Pneumonia | 131 | 49 |
| Femur fracture | 108 | 32 |
| Death | 88 | 10 |
| Pyrexia | 80 | 33 |
| Vomiting | 79 | 34 |
Showing 5 of 10 adverse events.
Which publications cover Genetic Diseases, X-Linked? (88)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Decoding VEXAS syndrome: emerging insights into pathogenesis and clinical management. | Current opinion in rheumatology | 2026 | Yes |
| Liver involvement in a large cohort of patients with erythropoietic protoporphyria or X-linked protoporphyria. | Hepatology communications | 2025 | Yes |
| Case report: anti-IL-6 autoantibodies in a patient with immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome. | Frontiers in immunology | 2025 | Yes |
| The International X-Linked Hypophosphatemia (XLH) Registry: first interim analysis of baseline demographic, genetic and clinical data. | Orphanet journal of rare diseases | 2023 | Yes |
| Long-term Burosumab Administration Is Safe and Effective in Adults With X-linked Hypophosphatemia. | The Journal of clinical endocrinology and metabolism | 2022 | Yes |
Showing 5 of 88 publications.
Sub-conditions in MeSH (25)
MeSH descendants one level down with at least one trial, most trials first.
+15 more sub-conditions
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: prevalence and rare-disease identity.